HUE035779T2 - Gene therapy for lysosomal storage disorders - Google Patents

Gene therapy for lysosomal storage disorders Download PDF

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Publication number
HUE035779T2
HUE035779T2 HUE08756596A HUE08756596A HUE035779T2 HU E035779 T2 HUE035779 T2 HU E035779T2 HU E08756596 A HUE08756596 A HU E08756596A HU E08756596 A HUE08756596 A HU E08756596A HU E035779 T2 HUE035779 T2 HU E035779T2
Authority
HU
Hungary
Prior art keywords
aav
brain
disease
transgene
acid
Prior art date
Application number
HUE08756596A
Other languages
English (en)
Hungarian (hu)
Inventor
James Dodge
Seng Cheng
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of HUE035779T2 publication Critical patent/HUE035779T2/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
HUE08756596A 2007-06-06 2008-06-02 Gene therapy for lysosomal storage disorders HUE035779T2 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US92494707P 2007-06-06 2007-06-06

Publications (1)

Publication Number Publication Date
HUE035779T2 true HUE035779T2 (en) 2018-05-28

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE08756596A HUE035779T2 (en) 2007-06-06 2008-06-02 Gene therapy for lysosomal storage disorders

Country Status (14)

Country Link
US (2) US8796236B2 (el)
EP (2) EP2158322B1 (el)
AR (1) AR070000A1 (el)
CY (1) CY1119295T1 (el)
DK (1) DK2158322T3 (el)
ES (2) ES2905616T3 (el)
HR (1) HRP20171165T1 (el)
HU (1) HUE035779T2 (el)
IL (3) IL202522A (el)
LT (1) LT2158322T (el)
PL (2) PL2158322T3 (el)
PT (2) PT3252161T (el)
SI (1) SI2158322T1 (el)
WO (1) WO2008154198A1 (el)

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ES2697502T3 (es) 2006-02-09 2019-01-24 Genzyme Corp Administración intraventricular lenta
PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8242086B2 (en) * 2008-11-12 2012-08-14 Duke University Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene
CA2759438C (en) 2009-04-30 2021-03-30 Fondazione Centro San Raffaele Del Monte Tabor Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
ES2745470T3 (es) * 2011-03-31 2020-03-02 Univ Iowa Res Found Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
SI2751279T1 (en) 2011-08-31 2018-01-31 St. Jude Children's Research Hospital Procedures and compositions for detecting the level of activity of lysosomal exocytosis and application procedures
BR112015028605A8 (pt) * 2013-05-15 2019-12-24 Univ Minnesota uso de uma composição e uso de um imunossupressor e uma composição
WO2015031686A1 (en) * 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
WO2016172155A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR20180098294A (ko) * 2015-12-15 2018-09-03 젠자임 코포레이션 뮤코지질증 ii형을 치료하기 위한 아데노-관련 바이러스 벡터
CN116440292A (zh) * 2016-01-19 2023-07-18 加利福尼亚大学董事会 Danon病和其它自噬障碍的治疗方法
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
AU2017355502B2 (en) * 2016-11-04 2023-08-31 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2018237066A1 (en) 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
KR20200104852A (ko) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2019222314A1 (en) * 2018-05-15 2019-11-21 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a
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WO2020072354A1 (en) * 2018-10-01 2020-04-09 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
JP2022525848A (ja) * 2019-02-26 2022-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クラッベ病の治療に有用な組成物
TW202104596A (zh) * 2019-04-12 2021-02-01 美商編碼治療公司 用於治療劑投予的組成物及方法
JP2023505851A (ja) 2019-12-10 2023-02-13 武田薬品工業株式会社 ハンター病治療用のアデノ随伴ウイルスベクター
JP2023523573A (ja) * 2020-04-14 2023-06-06 ジェネトン 酸性セラミダーゼ欠乏症を処置するためのベクター

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PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
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Also Published As

Publication number Publication date
WO2008154198A1 (en) 2008-12-18
IL252898B (en) 2019-06-30
US8796236B2 (en) 2014-08-05
EP3252161A1 (en) 2017-12-06
EP2158322A1 (en) 2010-03-03
EP2158322B1 (en) 2017-05-03
IL252898A0 (en) 2017-08-31
PT3252161T (pt) 2022-02-01
EP3252161B1 (en) 2021-11-17
PL2158322T3 (pl) 2017-10-31
IL266734B (en) 2021-10-31
AR070000A1 (es) 2010-03-10
SI2158322T1 (sl) 2017-10-30
DK2158322T3 (en) 2017-08-28
LT2158322T (lt) 2017-08-25
PT2158322T (pt) 2017-08-09
EP2158322A4 (en) 2011-06-01
HRP20171165T1 (hr) 2017-12-15
IL202522A0 (en) 2011-08-01
IL202522A (en) 2017-10-31
US20100173979A1 (en) 2010-07-08
ES2905616T3 (es) 2022-04-11
PL3252161T3 (pl) 2022-03-07
CY1119295T1 (el) 2018-02-14
IL266734A (en) 2019-07-31
ES2635726T3 (es) 2017-10-04
US11369693B2 (en) 2022-06-28
US20150151007A1 (en) 2015-06-04

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